FightMND Grant to Fund Phase 2 Study of Investigational Therapy for ALS

FightMND Grant to Fund Phase 2 Study of Investigational Therapy for ALS

Continuing its support for a potential therapy for amyotrophic lateral sclerosis (ALS), Australia-based organization FightMND has granted about $700,000 to biopharmaceutical company Collaborative Medicinal Development.

The money will fund a randomized, double-blind, placebo-controlled study of CuATSM, one of CMD’s lead investigational therapies. According to a news release, the Phase 2 study will begin later this year. Research details will soon be available at clinicaltrials.gov. The overall goal is to gain U.S. Food and Drug Administration (FDA) approval for the therapy.

“FightMND’s support for CMD to perform a Phase 2 study of CuATSM is consistent with our goals,”  said Rebecca Sheean, PhD, research manager for FightMND. “We are excited that our support for CMD has allowed CuATSM to advance toward the next phase of clinical testing.”

Established in 2014 to find treatments and a cure for motor neuron disease (MND), the nonprofit FightMND backed the medication’s Phase 1 trial three years ago.

The three-part trial (NCT02870634) showed that the once-daily oral medication may slow disease progression and improve patients’ respiratory and cognitive function. Studied in patients with sporadic and familial ALS, the investigation identified a Phase 2 recommended dose, examined CuATSM’s pharmacokinetic properties, confirmed treatment tolerability, and assessed the medication’s efficacy.

Data showed that after 24 weeks of CuATSM treatment at the recommended Phase 2 dose, ALS progression slowed markedly in those with sporadic ALS. Results also showed enhanced respiratory and cognitive ability. This is in line with preclinical studies in mouse models of ALS, which demonstrated that CuATSM performed dose-dependently when administered to animals.

The therapy is a small artificial molecule that can transport copper to cells containing damaged mitochondria, the cellular compartments that produce energy. Damaged mitochondria are widely considered a hallmark for ALS and other neurodegenerative diseases.

It is estimated that at least 30,000 people in the United States and 450,000 worldwide are living with ALS.

CMD, with bases in the United States and Australia, develops innovative therapies for neurodegenerative diseases.

 

3 comments

  1. jan berkhey says:

    nurown in israel seem to be the best therapie, I hope this treatment willbe very soon available for us. There are people dying, those people are very desperate!!!! Why does it take so long!!

  2. Robert L. Berman says:

    How can compassionate usage of this new drug be obtained? Or how can this drug be purchased off label?
    A Phase 2 trial, if successful, followed by a Phase 3 trial will take years to complete, if successful and attain FDA Approval. Many ALS patients do not have the
    advantage of those extra years. That’s why the “Right to Try” act was passed in the United States Congress and publicized by the President in a signing ceremony. There must be some way to obtain this potential medication without the intolerable waiting period; especially those who have nothing to lose and all to gain. Please respond to my email.

  3. • What about us living with als fighting to breathe, eat, walk and talk?
    •What about all the plans and dreams that ended with diagnosis of als?
    •We are beautiful hearts and have children that need us to be here to love them.
    •Falls may break bones and require stitches. But still we get back up and fight.
    •We have to make tough choices of ventilators, feeding tubes, rollators, braces, nursing homes to survive.
    •Since 1869 als has baffled neurologists and researchers all over the world.
    •Als is a disease we want cured, we looked to researchers to solved the puzzle.
    •What about Clinical trials that excludes us and take too long for those who have so little time left.
    •Sometimes we feel we were sold down the river. We are not invisible.
    •I participate in everything I can to help unlock the mysteries of als. I do believe there is promsing research, hopefully not 10 years down the road. I believe researchers are dedicated to finding a cure.
    •We pray to God for mankind to have the knowledge to find a cure. We look to the stars and pray to God that he will give us the strength inside to fight this cruel battle.
    •Als depletes you bank account. Insurances do not cover everything. You are not able to work. Social security barely covers the basic necessities of life.
    •Life is a daily struggle with ALS. I believe life is a precious gift. I love my family, life to much to give up. I keep hope alive.
    •I do not want als to take control of my body or my life. I challenge my mind to change the I can’t to I can.
    I put on my Queen of Denial Crown’. I am going to fake it until I make it. I am warrior; I will survive.
    •There needs to be changes.
    •Be a “light” to those who cannot help themselves.
    •Als affects 1 in 400 people. It affects all ages, all sexes, all races, all ages, rich or poor, famous or everyday person.
    Als is my abuser. It will silence my voice, will rob me of the ability to eat, will stop me in my steps, will take my breath, leave my body paralyzed and will steal my life. It could be you or someone you love.

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